公开(公告)号：WO2017194902A3

公开(公告)日：2018-01-18

申请号：PCT/FR2017051164

申请日：2017-05-12

Applicant: VECTALYS

Inventor： BOUILLE PASCALE ,  GAYON RÉGIS ,  LAMOUROUX LUCILLE ,  ICHE ALEXANDRA

IPC: C07K14/08 ,  C07K14/005 ,  C12N7/00 ,  C12N7/02 ,  C12N7/04 ,  C12N15/86 ,  C12N15/867 ,  C12N15/90

CPC classification number: C07K14/005 ,  C07K2319/735 ,  C07K2319/85 ,  C12N7/00 ,  C12N15/86 ,  C12N15/907 ,  C12N2740/16023 ,  C12N2740/16043 ,  C12N2740/16052 ,  C12N2795/18122 ,  C12N2800/24 ,  C12N2800/50

Abstract: The invention relates to a retroviral particle comprising a Gag polyprotein-derived protein, an envelope protein, optionally an integrase and at least two encapsidated non-viral RNAs, each of said encapsidated non-viral RNAs containing an RNA sequence of interest linked to an encapsidation sequence, each encapsidation sequence being recognized by a binding domain that is introduced into the Gag polyprotein-derived protein and/or into the integrase; furthermore, at least one of the sequences of interest of the encapsidated non-viral RNAs comprises a portion coding for a nuclease.

Abstract translation: 本发明涉及包含Gag多蛋白衍生蛋白，包膜蛋白，任选地整合酶和至少两种衣壳化的非病毒RNA的逆转录病毒颗粒，每种所述衣壳化的非病毒RNA含有连接至衣壳化的感兴趣的RNA序列 每个包壳序列被引入Gag多蛋白衍生蛋白和/或整合酶中的结合结构域识别; 此外，衣壳化的非病毒RNA的至少一个感兴趣的序列包含编​​码核酸酶的部分。

公开(公告)号：WO2015095167A2

公开(公告)日：2015-06-25

申请号：PCT/US2014070552

申请日：2014-12-16

Applicant: CHATTERJEE DEB K ,  KACZMARCZYK STANISLAW J

Inventor： CHATTERJEE DEB K ,  KACZMARCZYK STANISLAW J

IPC: A61K39/00

CPC classification number: C12N15/86 ,  A61K39/0011 ,  A61K39/12 ,  A61K45/06 ,  A61K2039/5258 ,  A61K2039/53 ,  A61K2039/585 ,  C12N7/00 ,  C12N2740/11023 ,  C12N2740/11042 ,  C12N2770/36123 ,  C12N2770/36134 ,  C12N2770/36143 ,  C12N2770/36145 ,  C12N2770/36152 ,  C12N2770/36171 ,  C12N2800/24 ,  C12N2810/6081

Abstract: Described herein is a method of preventing or treating a disease in a mammalian subject, comprising administering to the subject who is in need thereof an effective dosage of a pharmaceutical composition comprising a virus like particle (VLP) comprising: an alphavirus replicon comprising a recombinant polynucleotide, wherein the polynucleotide comprises a sequence encoding both subunits of a human class II major histocompatibility antigen, a retroviral gag protein, and a fusogenic envelope protein, wherein the VLP does not contain an alphavirus structural protein gene.

Abstract translation: 本文描述了一种预防或治疗哺乳动物受试者的疾病的方法，其包括向有需要的受试者施用有效剂量的包含病毒样颗粒（VLP）的药物组合物，其包含：包含重组多核苷酸的甲病毒复制子 其中所述多核苷酸包含编码人类II主要组织相容性抗原的两个亚基的序列，逆转录病毒gag蛋白和融合包膜蛋白，其中所述VLP不含有甲病毒属结构蛋白基因。

公开(公告)号：WO2013114199A8

公开(公告)日：2014-09-25

申请号：PCT/IB2013000198

申请日：2013-01-23

Applicant: BAYLOR COLLEGE MEDICINE

Inventor： LEE BRENDAN ,  GUSE KILIAN ,  RUAN ZHECHAO

IPC: C12N15/86 ,  A61K48/00

CPC classification number: C12N15/86 ,  A61K48/00 ,  C07K14/545 ,  C12N7/00 ,  C12N2710/10043 ,  C12N2710/10071 ,  C12N2710/10343 ,  C12N2710/10371 ,  C12N2800/24

Abstract: The invention relates to an adenoviral-based biological delivery and expression system for use in the treatment or prevention of osteoathritis in human or mammalian joints by long- term inducible gene expression of human or mammalian interleukin-1 receptor antagonist (II-1 Ra) in synovial cells, comprising a helper-dependent adenoviral vector containing a nucleic acid sequence encoding for human or mammalian interleukin-1 receptor antagonist (II-1 Ra), left and right inverted terminal repeats (L ITR and R ITR), the adenoviral packaging signal and non-viral, non-coding stuffer nucleic acid sequences, wherein the expression of the human or mammalian interleukin-1 receptor antagonist (II-1 Ra) gene within synovial cells is regulated by an inflammation-inducible promoter.

Abstract translation: 本发明涉及基于腺病毒的生物递送和表达系统，用于通过人或哺乳动物白细胞介素-1受体拮抗剂（II-1Ra）的长期诱导型基因表达来治疗或预防人或哺乳动物关节中的骨质炎 滑膜细胞，其包含辅助性依赖性腺病毒载体，其包含编码人或哺乳动物白细胞介素-1受体拮抗剂（II-1Ra）的核酸序列，左右反向末端重复（L ITR和R ITR），腺病毒包装信号 和非病毒的非编码填充核酸序列，其中滑膜细胞内的人或哺乳动物白细胞介素-1受体拮抗剂（II-1Ra）基因的表达由炎症诱导型启动子调节。

公开(公告)号：WO2014115022A1

公开(公告)日：2014-07-31

申请号：PCT/IB2014/000071

申请日：2014-01-27

Applicant: BAYLOR COLLEGE OF MEDICINE

Inventor： RUAN, Zhechao ,  GUSE, Kilian ,  LEE, Brendan

IPC: C12N15/86

CPC classification number: C12N15/86 ,  A61K48/0058 ,  C12N2710/10043 ,  C12N2710/10343 ,  C12N2800/24

Abstract: The present invention relates to gene therapy delivery and expression systems comprising at least one helper-dependent adenoviral vector containing a nucleic acid sequence encoding for proteoglycan 4 (PRG4) or a biologically active fragment thereof. The invention further relates to a pharmaceutical composition comprising a therapeutically effective amount of at least one helper-dependent adenoviral vector containing said nucleic acid sequence encoding for proteoglycan 4 (PRG4), or a homolog thereof from any other species, or a biologically active fragment thereof. The invention also relates to the use of the novel gene therapy delivery and expression system according to the invention for use in the prevention and/or treatment of camptodactyly-arthropathy-coxa vara-pericarditis (CACP), or a musculoskeletal disorder such as a joint disorder or joint disease.

Abstract translation: 本发明涉及包含至少一种辅助依赖性腺病毒载体的基因治疗递送和表达系统，其包含编码蛋白聚糖4（PRG4）的核酸序列或其生物活性片段。 本发明还涉及药物组合物，其包含治疗有效量的至少一种辅助依赖性腺病毒载体，其含有编码蛋白聚糖4（PRG4）的核酸序列，或其任何其他物种的同源物或其生物活性片段 。 本发明还涉及根据本发明的新型基因治疗递送和表达系统用于预防和/或治疗营养不良 - 关节病 - 龋齿心包炎（CACP）或肌肉骨骼疾病如关节的用途 无序或关节疾病。

公开(公告)号：WO2013181440A1

公开(公告)日：2013-12-05

申请号：PCT/US2013/043433

申请日：2013-05-30

Applicant: BAYLOR COLLEGE OF MEDICINE ,  UNIVERSITY OF WASHINGTON

Inventor： ZECHIEDRICH, Lynn, E. ,  FOGG, Jonathan ,  CATANESE, JR., Daniel, James ,  BAKKALBASI, Erol ,  MAIZEL, Nancy ,  HUMBERT, Olivier

IPC: A61K48/00

CPC classification number: C12N15/85 ,  A61K48/005 ,  C12N15/10 ,  C12N15/8213 ,  C12N15/907 ,  C12N2800/24 ,  C12N2800/30 ,  C12N2800/80

Abstract: In some embodiments the present disclosure provides a composition for targeted alteration of a DNA sequence and methods of altering the targeted DNA sequence using the composition. In some embodiments such a composition comprises a MiniVector comprising a nucleic acid sequence template for homology-directed repair, alteration, or replacement of the targeted DNA sequence within a cell in vivo or in vitro, where the MiniVector lacks both a bacterial origin of replication and an antibiotic selection gene, and wherein the Mini Vector has a size up to about 2,500 base pairs.

Abstract translation: 在一些实施方案中，本公开提供了用于靶向改变DNA序列的组合物和使用该组合物改变靶DNA序列的方法。 在一些实施方案中，这样的组合物包含MiniVector，其包含用于在体内或体外在细胞内进行同源性修复，改变或置换靶DNA的序列的核酸序列模板，其中MiniVector缺少细菌复制起点， 一种抗生素选择基因，其中该迷你载体具有高达约2,500个碱基对的大小。

公开(公告)号：WO2013178344A1

公开(公告)日：2013-12-05

申请号：PCT/EP2013/001547

申请日：2013-05-24

Applicant: AMVAC AG

Inventor： WIEGAND, Marian

IPC: C12N15/86 ,  C12N5/071

CPC classification number: C12N15/86 ,  A61K48/00 ,  C07K14/4702 ,  C12N5/0696 ,  C12N7/00 ,  C12N2760/18822 ,  C12N2760/18843 ,  C12N2760/18852 ,  C12N2760/18862 ,  C12N2800/24

Abstract: The present invention provides a method of expressing at least one heterologous nucleic acid sequence in a cell, the method comprising introducing at least one heterologous nucleic acid sequence into a cell by infecting said cell with a recombinant negative-strand RNA virus vector comprising said at least one heterologous nucleic acid sequence, wherein the recombinant negative- strand RNA virus vector includes a viral genome coding for a mutated P protein, which leads to a loss of the viral genome replication ability without a loss of the viral transcription ability, and wherein said at least one heterologous nucleic acid sequence encodes a cellular reprogramming or programming factor or a therapeutic protein. In addition, the present invention provides a cell or a population of cells prepared in vitro by said method as well as a pharmaceutical composition comprising said cell or population of cells.

Abstract translation: 本发明提供了在细胞中表达至少一种异源核酸序列的方法，所述方法包括通过用重组负链RNA病毒载体感染所述细胞将至少一种异源核酸序列引入细胞，所述重组负链RNA病毒载体包含至少 一个异源核酸序列，其中重组负链RNA病毒载体包括编码突变的P蛋白的病毒基因组，其导致病毒基因组复制能力的丧失而不损失病毒转录能力，并且其中所述 至少一个异源核酸序列编码细胞重编程或编程因子或治疗性蛋白质。 此外，本发明提供了通过所述方法体外制备的细胞或细胞群以及包含所述细胞或细胞群的药物组合物。

公开(公告)号：WO2012170911A2

公开(公告)日：2012-12-13

申请号：PCT/US2012/041693

申请日：2012-06-08

Applicant: BLUEBIRD BIO, INC. ,  DENARO, Maria, Joann ,  FINER, Mitchell, Howard ,  VERES, Gabor

Inventor： DENARO, Maria, Joann ,  FINER, Mitchell, Howard ,  VERES, Gabor

CPC classification number: A61K35/51 ,  A61K35/28 ,  A61K38/177 ,  A61K48/005 ,  A61K48/0058 ,  C07K14/705 ,  C12N5/0686 ,  C12N7/00 ,  C12N15/86 ,  C12N2740/15043 ,  C12N2740/16043 ,  C12N2800/24 ,  C12N2830/00 ,  C12N2830/008 ,  C12N2830/50 ,  C12N2830/60 ,  C12N2840/00

Abstract: The present invention provides compositions ccomprising retroviral vectors, transduced cells, and methods of using the same for gene therapy. In particular, the present invention relates to lentiviral vectors and cells transduced with those vectors to provide gene therapy to subjects having an adrenoleukodystrophy and/or adrenomyeloneuropathy.

Abstract translation: 本发明提供了包含逆转录病毒载体，转导细胞的组合物，以及将其用于基因治疗的方法。 特别地，本发明涉及慢病毒载体和用这些载体转导的细胞，以向具有肾上腺脑白质营养不良和/或肾上腺皮质神经病变的受试者提供基因治疗。

公开(公告)号：WO2012141653A1

公开(公告)日：2012-10-18

申请号：PCT/SE2012/050407

申请日：2012-04-13

Applicant: NAUCLÉR, Cecilia

Inventor： NAUCLÉR, Cecilia

IPC: C07K14/045 ,  G01N33/68

CPC classification number: C12N7/00 ,  C07K14/005 ,  C12N2710/16121 ,  C12N2710/16122 ,  C12N2800/24 ,  C12Q1/701 ,  G01N33/5091 ,  G01N33/56994 ,  G01N33/57407 ,  G01N2333/045 ,  G01N2800/50

Abstract: The present invention relates to a genetic variant of CMV, said genetic variant lacking intron 2 of the IE region of CMV (CMV ΙΕΔi2). The present invention also related to various uses of this genetic variant as well as RNA splice variants transcribed therefrom, and proteins expressed from the RNA splice variants, such as in the diagnosis of a CMV related cancer disease, and identification of individuals at risk of developing cancer or risk of transferring the CMV ΙΕΔi2 virus with a human sample and prevention and treatment through targeting of unique CMV IE proteins for immunotherapy and vaccination.

Abstract translation: 本发明涉及CMV的遗传变体，所述遗传变体缺乏CMV（CMV2i2）的IE区域的内含子2。 本发明还涉及该遗传变异体的各种用途以及从其转录的RNA剪接变体，以及从RNA剪接变体表达的蛋白质，例如诊断CMV相关的癌症疾病，以及鉴定处于发展风险中的个体 癌症或将CMV？i2病毒转染人类样本的风险，并通过靶向独特的CMV IE蛋白进行免疫治疗和疫苗接种来进行预防和治疗。

公开(公告)号：WO2012085282A1

公开(公告)日：2012-06-28

申请号：PCT/EP2011/073984

申请日：2011-12-23

Applicant: MOLOGEN AG ,  SCHROFF, Matthias ,  KLEUSS, Christiane ,  KAPP, Kerstin

Inventor： SCHROFF, Matthias ,  KLEUSS, Christiane ,  KAPP, Kerstin

IPC: C12N15/11 ,  C12N15/09 ,  C12N15/85

CPC classification number: C12N15/85 ,  A61K48/00 ,  C12N2800/24

Abstract: The invention relates to a minimalistic gene expression construct, its transfer into cells and its use for gene expression for molecular-medical applications. According to the disclosure, a DNA construct for gene expression is provided, wherein the construct is a linear and open-chained DNA double strand comprising a promoter sequence, a coding sequence and a termination signal, wherein the construct comprises at least one L-DNA nucleotide.

Abstract translation: 本发明涉及简约基因表达构建体，其转移到细胞中及其用于分子医学应用的基因表达的用途。 根据本公开，提供了用于基因表达的DNA构建体，其中所述构建体是包含启动子序列，编码序列和终止信号的线性和开链DNA双链，其中所述构建体包含至少一个L-DNA 核苷酸。

公开(公告)号：WO2010094280A1

公开(公告)日：2010-08-26

申请号：PCT/DE2010/075012

申请日：2010-02-05

Applicant: CEVEC PHARMACEUTICALS GMBH ,  SCHIEDNER, Gudrun ,  VOLPERS, Christoph

Inventor： SCHIEDNER, Gudrun ,  VOLPERS, Christoph

IPC: C12N5/079

CPC classification number: C12N5/0605 ,  C12N15/85 ,  C12N2510/04 ,  C12N2800/24

Abstract: Die vorliegende Erfindung betrifft eine permanente humane Zelllinie, umfassend die Nukleinsäuresequenzen für die adenoviralen Genfunktionen E1A und E1B und die Nukleinsäuresequenz für das grosse T-Antigen von SV40 oder das Epstein-Barr-Virus (EBV) Nuclear Antigen-1 (EBNA-1). Die vorliegende Erfindung betrifft ferner ein Verfahren zur transienten Expression rekombinanter Polypeptide und Proteine in der permanenten humanen Zelllinie.

Abstract translation: 本发明涉及包含所述腺病毒基因功能E1A和E1B和SV40或Epstein-Barr病毒（EBV）核抗原1（EBNA-1）的大T抗原的核酸序列的核酸序列的永久人细胞系。 本发明还涉及一种用于重组多肽和蛋白质在所述持久性人细胞系的瞬时表达的方法。